P1497: BURDEN OF SICKLE CELL DISEASE: RESULTS FROM THE REAL WORLD ASSESSMENT SURVEY FOR SICKLE CELL DISEASE IN SAUDI (ROARS)
Authors: W. Jastaniah, A. Al Zayed, H. Al Saeed, M. AlDarwish, M. Albagshi, H. Malhan, A. Tarawa, A. AlManea, I. AlHazmi, M. Qari, A. Al Jefr, A. Alzahrani, M. Alzahran, W. Soliman, F. Askar, H. Ezzat
Journal: HemaSphere (2022)

Background: The Kingdom of Saudi Arabia (KSA) has one of the highest prevalence of sickle cell disease (SCD; an inherited hemoglobinopathy) in the world, with up to 2.6% of the population affected. The severity of SCD in KSA varies widely, likely due to existing haplotype variations. This creates a unique opportunity to understand the impact of SCD on patients' (pts') quality of life (QoL) and available treatments, and to inform management strategies that can reduce disease burden and improve QoL. Aims: The Real World Assessment Survey for Sickle Cell Disease in Saudi (ROARS) is a cross-sectional survey of pts with SCD and treating healthcare professionals (HCPs) which was designed to understand the burden of SCD, including QoL, symptoms, treatment strategies, treatment goals, and management approaches, in KSA. Methods: Between September and December 2021, pts with SCD (age: 5-65 years) and HCPs (>2 years SCD experience, SCD load >50 pts, >50% of practice time spent treating SCD pts) were surveyed (in person or via caregiver if pts were between 5-14 years) using the structured, bilingual ROARS questionnaires (English/Arabic) through personal quantitative web-assisted telephone interviews. ROARS, developed by SCD experts from KSA and Novartis, includes the global health domain of Patient-Reported Outcomes Measurement Information System (PROMIS), a validated tool for assessing QoL among SCD pts and caregivers of different age ranges. Questions covered topics such as disease impact on QoL, symptoms, disease complications, treatment goals, and treatment strategies. Results: Overall, 1011 pts (pediatric: n=504; adult: n=507) and 64 HCPs (specialists: n=31; non-specialists: n=33) completed the survey. The mean (standard deviation; SD) age was 20.6 (13.2) years, 52% of pts were female, and 60% were homozygous for hemoglobin S (self-reported genotype). Data were collected from 11 cities, with 90% of respondents from Eastern and South-Western provinces of KSA where prevalence of SCD is the highest. Burden of the disease was significant regardless of the region; QoL was rated as poor/fair by 27% of adult and 38% of pediatric pts (Figure). Pts reported that SCD caused a negative impact on emotions (28%), school achievement (19%), and a reduction in work hours (16%). The top 5 symptoms reported by both pediatric and adult pts were bone ache (83%), fatigue/tiredness (68%), headache (56%), poor appetite (47%), and anxiety (45%). The mean (SD) incidence of vaso-occlusive crises (VOCs) in the 12 months prior to survey completion was 6.5 (4.6) and severe VOCs resulting in heathcare facility visits was 5.5 (4.3). Reducing the number of VOCs and increasing the number of days free of severe pain was an important treatment goal in 84% of pediatric pts and in 71% of adult pts. Overall, 51% of pts managed their VOCs at home. The most important factors in treatment decision were improving QoL and preventing end organ damage, reported by 56% and 45% of HCPs, respectively. Top treatments that the pts are currently receiving are antibiotics (48%), opioids (33%), hydroxyurea (33%), blood transfusion (31%), and iron chelation therapy (26%). Innovative treatments were reported by 5% of pts and hematopoietic stem cell transplantation in only 2%. Overall, 68% of pts reported dissatisfaction with their current treatments for SCD. Image:Summary/Conclusion: Based on ROARS data, which incorporated pts' voices, there is a significant burden of SCD among pts in KSA. This study highlights the need for national-specific plans for effective management strategies and allocation of resources.